Health state utility values for metastatic pancreatic cancer using a composite time trade-off based on the vignette-based approach in Japan

Backgrounds Limited information is available on the utility values of metastatic pancreatic cancer, focusing on different health statuses, selected chemotherapy, and related grades 1/2 and 3/4 adverse events (AEs). We evaluated Japanese societal-based health-related utility values for metastatic pancreatic cancer by considering different grade toxicities commonly associated with chemotherapy using the vignette-based method. Methods We developed health status scenarios for patients with metastatic pancreatic cancer undergoing chemotherapy and conducted utility research using the developed scenarios in four steps: ‘literature review,’ ‘exploratory interview,’ ‘content validation’, and ‘utility research’. In the development process, to consider the impact of AEs of chemotherapy for metastatic pancreatic cancer on health state utility values, we selected neutropenia, febrile neutropenia, diarrhea, nausea and vomiting, and neuropathy as representative AEs. Each AE was classified as either grade 1/2 or 3/4. We confirmed our created scenarios through cognitive interviews with the general population and clinical experts to validate the content. Finally, we developed 11 scenarios for using ‘utility research,’ evaluated in a societal-based valuation study using the face-to-face method. Participants for ‘utility research’ were the general population, and they evaluated these scenarios in the composite time trade-off (cTTO) and visual analog scale (VAS) of the European quality of life (EuroQol) valuation technology to derive health state utility scores. Results Of 220 responders who completed this survey, 201 were adapted into the analysis population. Stable disease with no AEs (reference state) had a mean utility value of 0.653 using cTTO. The lowest mean utility score in the stable state was 0.242 (stable disease + grade 3/4 vomiting). VAS results ranged from 0.189 to 0.468, depending on the various grades of AEs in stable disease. In addition, grade 3/4 AEs and grade 1/2 nausea/vomiting were associated with significantly greater disutility. Utility values were also strongly influenced by the direct impact of AE on physical symptoms, severity and their experience. In addition, 95.9% of the respondents agreed that they understood the questions in the post-response questionnaire. Conclusions We clarified the health state utility values of patients with metastatic pancreatic cancer based on the general population in Japan. The effect on utilities should be considered not only for serious AEs, but also for minor AEs. Supplementary Information The online version contains supplementary material available at 10.1186/s13561-022-00413-8.


Mental
You are very anxious about the possibility of dying from the disease.
Adverse effects None.
2 SD + Neutropenia Grade1/2 Summary You have a life-threatening disease, but his condition has been stabilized by treatment.

Physical symptoms
Appetite You do not have enough appetite to lose weight.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As an adverse event of treatment, you have a mild decrease in immunity, but you do not have any physical symptoms such as lethargy or fever.

SD + Neutropenia
Grade3/4 Summary You have a life-threatening disease, but his condition has been stabilized by treatment.

Physical symptoms
Appetite You do not have enough appetite to lose weight.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As an adverse event of treatment, your immune system is compromised. You do not have any physical symptoms such as lethargy or fever, but you have to refrain from going out due to the increased risk of infection.
You have a life-threatening disease, but his condition has been stabilized by treatment.

Physical symptoms
Appetite You do not have enough appetite to lose weight.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As the adverse event of treatment, you have lethargy and a fever over 38 degrees, requiring urgent hospitalization (intravenous fluids, etc.).

Summary
You have a life-threatening disease, but his condition has been stabilized by treatment.

Physical symptoms
Appetite You do not have enough appetite to lose weight.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As an adverse event of treatment, you have 4 to 6 more bowel movements and diarrhea per day than usual.
You have a life-threatening disease, but his condition has been stabilized by treatment.

Physical symptoms
Appetite You do not have enough appetite to lose weight.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As an adverse event of treatment, you have diarrhea more than seven times a day, which requires hospitalization (intravenous fluids, etc.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As an adverse event of treatment, you have persistent tingling pain and numbness in your hands and feet, which is uncomfortable.

SD + Neuropathy
Grade3/4 Summary You have a life-threatening disease, but his condition has been stabilized by treatment.

Physical symptoms
Appetite You do not have enough appetite to lose weight.

Fatigue
You are more tired than when you were healthy.
Body aches Sometimes you have strong pain. You take medication to relieve it.

Daily life at home and outside
You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You are very anxious about the possibility of dying from the disease.

Adverse effects
As an adverse event of treatment, you have severe numbness in your hands and feet, making it difficult to button or grip small objects, and when it is terrible, it is difficult to walk.

Summary
You have a life-threatening disease. We have tried everything to treat you, but your condition is worsening.

Adverse effects
None.
As an adverse event of treatment, you have nausea and vomiting 2~3 times a day.

Daily life at home and outside
You can live freely on your own. You can live by yourself at home, but you can only go out for a couple of hours.

Mental
You have no worries or concerns about your health.
You are very anxious about the possibility of dying from the disease.

Physical symptoms Appetite
You have the same appetite as usual. You do not have enough appetite to lose weight.

Fatigue
You can eating as usual.
You are more tired than when you were healthy.

Body aches
You have not pain. Sometimes you have strong pain. You take medication to relieve it.

Summary
Physically and mentally, the healthiest state.
You have a life-threatening disease, but his condition has been stabilized by treatment.
Case 1: Live only 10 years in "perfect health".
Case 1 and Case 2 are the same for you.
Case 2: Live for 10 years in "perfect health" and then live for 10 years in the "specific health condition" described above.

Case 1
Case 2 10 year 10 year 10 year QX You will be asked to think about the "Full health" and the "Impaired health" shown below.
After reading all the statements on the screen, imagine how you would feel if you were in such a health condition.

Full health
Full health Impaired health

QX_yy
We created two cases by combining two health conditions and years of survival.
When do you imagine the two cases in yourown life, which one you think is more preferable for you,Case 1 or Case 2, as shown below? eFigure 2: Distribution of utility values based on cTTO for SD and PD scenarios cTTO, composite time trade-off; F2F: face-to-face; SD: stable disease; PD: progressive disease; QOL, quality of life